News

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

CGTLive takes a look at recent progress in cell therapy development targeting solid tumors, featuring expert commentary on these advances.

Review top news and interview highlights from the week ending April 14, 2023.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed past, current, and future approaches to diagnosing and treating neuromuscular diseases.

Cellectis noted that the allogeneic product was manufactured at its new in-house facility in Paris.

Matthew Gornet, MD, spine surgeon from The Orthopedic Center of St. Louis discussed data seen with IDCT cell therapy.

Vigil cell therapy previously showed some efficacy as a monotherapy in patients with ovarian cancer.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the trend towards gene-targeted approaches in the neuromuscular disease space.

The CaMMouflage trial dosed its first participant in March 2023.

REGENXBIO’s RGX-202 previously received orphan drug and rare pediatric disease designations for DMD in late 2021 and early 2022, respectively.

The assistant professor of medicine at Vanderbilt University Medical Center discussed the work that has been done in the field in the past 10 years and work that still remains to be done.

Verismo Therapeutics’ SynKIR-110 previously received orphan drug designation from the FDA.

Immatics is prioritizing its TCR-T and TCER cell therapy programs and leaving behind its first-generation programs.

The executive vice president and chief research officer of MDA discussed the barriers to FDA approval currently facing those developing treatments for ultra-rare diseases.

The company’s pipeline is down to 1 remaining clinical-stage program that is enrolling in its first-in-human trial.

The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c

Review top news and interview highlights from the week ending April 7, 2023.

The trial’s dose-escalation cohorts are assessing results from patients with type 2a and type 1 diseases separately.

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.

Although the TCB-202-001 trial ended prematurely due to the COVID-19 pandemic, promising results warrant a phase 2 trial.

The spine surgeon from The Orthopedic Center of St. Louis discussed recent data on IDCT from the phase 1/2 DGX-A01 study.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Investigator Matthew Charman, PhD, told CGTLive™ that the new knowledge of the viral replication process could potentially inform the development of future gene therapies.

The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.

No treatment-emergent adverse events experienced were deemed related to AstroRx, although 9 serious TEAEs did occur.

Atsena Therapeutics’ AAV gene therapy is being evaluated in 15 patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D across 5 cohorts of various doses. Additional data are expected to be presented later this year.

The neurology resident at Penn Medicine spoke about the preparations taken in the lead up to tofersen’s PDUFA date, which is set for April 25, 2023.